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Big Data Research in Neuro-Ophthalmology

Promises and Pitfalls

Moss, Heather E. MD, PhD; Joslin, Charlotte E. OD, PhD; Rubin, Daniel S. MD; Roth, Steven MD

Section Editor(s): Costello, Fiona MD, FRCP(C); Prasad, Sashank MD

doi: 10.1097/WNO.0000000000000751
State-of-the-Art Review

Background: Big data clinical research involves application of large data sets to the study of disease. It is of interest to neuro-ophthalmologists but also may be a challenge because of the relative rarity of many of the diseases treated.

Evidence Acquisition: Evidence for this review was gathered from the authors' experiences performing analysis of large data sets and review of the literature.

Results: Big data sets are heterogeneous, and include prospective surveys, medical administrative and claims data and registries compiled from medical records. High-quality studies must pay careful attention to aspects of data set selection, including potential bias, and data management issues, such as missing data, variable definition, and statistical modeling to generate appropriate conclusions. There are many studies of neuro-ophthalmic diseases that use big data approaches.

Conclusions: Big data clinical research studies complement other research methodologies to advance our understanding of human disease. A rigorous and careful approach to data set selection, data management, data analysis, and data interpretation characterizes high-quality studies.

Departments of Ophthalmology and Neurology & Neurological Sciences (HEM), Stanford University, Palo Alto, California; Departments of Ophthalmology and Visual Sciences (CEJ), University of Illinois, School of Public Health, College of Medicine, Epidemiology and Public Health, Chicago, Illinois; Department of Anesthesia and Critical Care (DSR), University of Chicago, Chicago, Illinois; and Departments of Anesthesiology, Ophthalmology and Visual Sciences (SR), College of Medicine, University of Illinois, Chicago, Illinois.

Address correspondence to Heather E. Moss, MD, PhD, Department of Ophthalmology, Stanford University, Spencer Center for Vision Research, 2370 Watson Court, MC 5349, Palo Alto, CA 94303; E-mail:

National Institutes of Health (Bethesda, MD) grants R21 EY027447 (to S. Roth), K23 EY 024345 (to Moss), P30 EY 026877 to the Department of Ophthalmology at Stanford University, P30 EY001792 to the Department of Ophthalmology at the University of Illinois at Chicago, unrestricted grants from Research to Prevent Blindness, Inc. (New York, NY) to the Stanford Department of Ophthalmology and to the University of Illinois at Chicago Department of Ophthalmology and Visual Sciences, and by the Michael Reese Foundation (Chicago, IL) Pioneers Award to Dr. Roth.

The authors report no conflicts of interest.

© 2019 by North American Neuro-Ophthalmology Society